CCHSDyspnOndineNCT04447196
Prevalence of Rest Dyspnea and Impact of Non Invasive Ventilation on Breathing Sensations in CCHS Patients
An observational study of CCHS and Congenital Central Hypoventilation Syndrome, run by Association pour le Développement et l'Organisation de la Recherche en Pneumologie et sur le Sommeil at 1 site in France.
Trial summary
Congenital central hypoventilation syndrome (CCHS) is a neuro-respiratory disease characterized by lifethreatening sleep-related hypoventilation involving an alteration of CO2/H+ chemosensitivity. This suggests cortical activity during awakening to maintain breathing. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- CCHS, Congenital Central Hypoventilation Syndrome, Dyspnea
- What is being tested?
- MDP
- How many people
- 20 participants
- Where
- 1 siteFrance
- Study dates
- 15 Dec 2021to 1 Oct 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Adults with CCHS who already require long terme NIV but not phrenic nerve stimulation
Who may be able to join
- patients with CCHS confirmed by genetic test
- patients with long term non invasive ventilation
- no recent condition that could impact breathing sensations such as infection, pulmonary embolism, heart failure
Who cannot join
- pregnancy
- phrenic nerve stimulation
Trial locations and contacts
1 site in 1 country.
France1 site
INSERM-UPC UMR_S 1158, Service de Pneumologie et Réanimation, GH Pitié-Salpêtrière
Paris, 75013
About this study
Official title: Prevalence of Rest Dyspnea and Impact of Non Invasive Ventilation on Breathing Sensations in Patients With Congenital Central Hypoventilation Syndrome
Congenital central hypoventilation syndrome (CCHS) is a neuro-respiratory disease characterized by lifethreatening sleep-related hypoventilation involving an alteration of CO2/H+ chemosensitivity. This suggests cortical activity during awakening to maintain breathing. Cortical activity to keep breathing is usually associated with breathing discomfort ; this is the case in healthy subjects under non invasive ventilation (NIV) or with expiratory charge as well as in patients with amyotrophic lateral sclerosis. This can suggest that CCHS may be breathless at rest and this discomfort could be reduced by NIV.
The objective is to evaluate dyspnea with a multi dimensional score, MDP, in CCHS patient at rest in every day life and during 1H session of NIV.
Read more
The investigators will perform a prospective, including 20 CCHS patients. MDP scores will be measure before and after 1H-non invasive ventilation as well as a visual scale of 100mm in order to evaluate variation of breathing discomfort before/after NIV.
The investigators expect that CCHS patients don't have rest dyspnea but NIV would improve breathing discomfort that would mean they have latent rest dyspnea.
- Conditions
- CCHS, Congenital Central Hypoventilation Syndrome, Dyspnea
- Being tested
- MDP
- Medical subject headings
- Congenital central hypoventilation syndrome, Dyspnea
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MDPOther
Multi dimensional dyspnea profile scores at rest and during non invasive ventilation
| Study group | Role | What they receive |
|---|---|---|
| CCHS | 20 patients presenting with central hypoventilation syndrome during spontaneous breathing and with Non Invasive Ventilation Receives: Other: MDP |
What is being measured
The primary outcome is the main question the study is designed to answer.
"Immediate unpleasantness" corresponding to A.1 item of the multidimensional dyspnea profile
Measure MDP scores before and after NIV
Measured: Change in MDP score between baseline and after 1 hour of NIV
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jun 2020
- Study start15 Dec 2021 (estimated)
- Primary completion1 Oct 2022 (estimated)
- Study completion1 Oct 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CCHSStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Association pour le Développement et l'Organisation de la Recherche en Pneumologie et sur le Sommeil
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04447196
- Sponsor's protocol ID
- 2020-A00215-34
- Acronym
- DyspnOndine
- Status last verified
- Jul 2021
- First submitted
- 30 May 2020
- Last updated
- 12 Jul 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Jul 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prevalence of Rest Dyspnea and Impact of Non Invasive Ventilation on Breathing Sensations in Patients With Congenital Central Hypoventilation Syndrome
- ClinicalTrials.gov ID
- NCT04447196
- Acronym / sponsor ID
- DyspnOndine / 2020-A00215-34
- Conditions
- CCHS, Congenital Central Hypoventilation Syndrome, Dyspnea
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Association pour le Développement et l'Organisation de la Recherche en Pneumologie et sur le SommeilUniversity, hospital or other
- Study start
- 15 Dec 2021 (estimated)
- Primary completion
- 1 Oct 2022 (estimated)
- Study completion
- 1 Oct 2022 (estimated)
- First posted
- 25 Jun 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Congenital central hypoventilation syndrome (CCHS) is a neuro-respiratory disease characterized by lifethreatening sleep-related hypoventilation involving an alteration of CO2/H+ chemosensitivity. This suggests cortical activity during awakening to maintain breathing. Cortical activity to keep breathing is usually associated with breathing discomfort ; this is the case in healthy subjects under non invasive ventilation (NIV) or with expiratory charge as well as in patients with amyotrophic lateral sclerosis. This can suggest that CCHS may be breathless at rest and this discomfort could be reduced by NIV.
The objective is to evaluate dyspnea with a multi dimensional score, MDP, in CCHS patient at rest in every day life and during 1H session of NIV.
The investigators will perform a prospective, including 20 CCHS patients. MDP scores will be measure before and after 1H-non invasive ventilation as well as a visual scale of 100mm in order to evaluate variation of breathing discomfort before/after NIV.
The investigators expect that CCHS patients don't have rest dyspnea but NIV would improve breathing discomfort that would mean they have latent rest dyspnea.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CCHS | – | Other: MDP20 patients presenting with central hypoventilation syndrome during spontaneous breathing and with Non Invasive Ventilation |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adults with CCHS who already require long terme NIV but not phrenic nerve stimulation
Key inclusion criteria (3 listed)
- patients with CCHS confirmed by genetic test
- patients with long term non invasive ventilation
- no recent condition that could impact breathing sensations such as infection, pulmonary embolism, heart failure
Key exclusion criteria (2 listed)
- pregnancy
- phrenic nerve stimulation
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MDPMulti dimensional dyspnea profile scores at rest and during non invasive ventilation | Other | CCHS |
Primary outcome
"Immediate unpleasantness" corresponding to A.1 item of the multidimensional dyspnea profile
Time frame: Change in MDP score between baseline and after 1 hour of NIV
Measure MDP scores before and after NIV
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jul 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of CCHS and Congenital Central Hypoventilation Syndrome, run by Association pour le Développement et l'Organisation de la Recherche en Pneumologie et sur le Sommeil at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 12 Jul 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact MARLENE BRET (0698674449, marlene.bret@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
"Immediate unpleasantness" corresponding to A.1 item of the multidimensional dyspnea profile (time frame: Change in MDP score between baseline and after 1 hour of NIV). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Jul 2021.